✉news ScienceBiology first seen 1 d ago, last 1 d ago, peak #31
Engineered tRNAs Restore Full-Length Dystrophin in Mice
Researchers report that engineered transfer RNAs restored production of full-length dystrophin in a mouse model of Duchenne muscular dystrophy. The approach, known as suppressor tRNA therapy, reads through premature stop mutations that cut the muscle protein short. In treated mice, dystrophin was produced at levels that could point toward a treatment strategy for patients whose disease is driven by nonsense mutations, though human testing remains ahead.
Why now: A potential gene-therapy advance toward treating Duchenne muscular dystrophy has just been reported.
dystrophinengineered tRNAsDuchenne muscular dystrophymice
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